Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes
Identifieur interne : 001B18 ( Ncbi/Merge ); précédent : 001B17; suivant : 001B19Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes
Auteurs : Vanessa Borges Pires [Portugal] ; Ricardo Sim Es [Portugal] ; Kamel Mamchaoui [France] ; Célia Carvalho [Portugal] ; Maria Carmo-Fonseca [Portugal]Source :
- PLoS ONE [ 1932-6203 ] ; 2017.
Descripteurs français
- KwdFr :
- Dystrophine (biosynthèse), Dystrophine (génétique), Exons, Fibres musculaires squelettiques (métabolisme), Humains, Isoformes de protéines (biosynthèse), Isoformes de protéines (génétique), Lignée cellulaire, Myopathie de Duchenne (), Myopathie de Duchenne (génétique), Myopathie de Duchenne (métabolisme), Oligonucléotides (génétique), Oligonucléotides (usage thérapeutique), Oligonucléotides antisens (génétique), Oligonucléotides antisens (usage thérapeutique), Thérapie génétique (), Épissage des ARN.
- MESH :
- biosynthèse : Dystrophine, Isoformes de protéines.
- génétique : Dystrophine, Isoformes de protéines, Myopathie de Duchenne, Oligonucléotides, Oligonucléotides antisens.
- métabolisme : Fibres musculaires squelettiques, Myopathie de Duchenne.
- usage thérapeutique : Oligonucléotides, Oligonucléotides antisens.
- Exons, Humains, Lignée cellulaire, Myopathie de Duchenne, Thérapie génétique, Épissage des ARN.
English descriptors
- KwdEn :
- Cell Line, Dystrophin (biosynthesis), Dystrophin (genetics), Exons, Genetic Therapy (methods), Humans, Muscle Fibers, Skeletal (metabolism), Muscular Dystrophy, Duchenne (genetics), Muscular Dystrophy, Duchenne (metabolism), Muscular Dystrophy, Duchenne (therapy), Oligonucleotides (genetics), Oligonucleotides (therapeutic use), Oligonucleotides, Antisense (genetics), Oligonucleotides, Antisense (therapeutic use), Protein Isoforms (biosynthesis), Protein Isoforms (genetics), RNA Splicing.
- MESH :
- chemical , biosynthesis : Dystrophin, Protein Isoforms.
- chemical , genetics : Dystrophin, Oligonucleotides, Oligonucleotides, Antisense, Protein Isoforms.
- genetics : Muscular Dystrophy, Duchenne.
- metabolism : Muscle Fibers, Skeletal, Muscular Dystrophy, Duchenne.
- methods : Genetic Therapy.
- chemical , therapeutic use : Oligonucleotides, Oligonucleotides, Antisense.
- therapy : Muscular Dystrophy, Duchenne.
- Cell Line, Exons, Humans, RNA Splicing.
Abstract
Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.
Url:
DOI: 10.1371/journal.pone.0181065
PubMed: 28742140
PubMed Central: 5524367
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PMC:5524367Le document en format XML
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<term>Genetic Therapy (methods)</term>
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<term>Muscle Fibers, Skeletal (metabolism)</term>
<term>Muscular Dystrophy, Duchenne (genetics)</term>
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<front><div type="abstract" xml:lang="en"><p>Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.</p>
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<double pmid="28742140"><pmc><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes</title>
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<author><name sortKey="Sim Es, Ricardo" sort="Sim Es, Ricardo" uniqKey="Sim Es R" first="Ricardo" last="Sim Es">Ricardo Sim Es</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Mamchaoui, Kamel" sort="Mamchaoui, Kamel" uniqKey="Mamchaoui K" first="Kamel" last="Mamchaoui">Kamel Mamchaoui</name>
<affiliation wicri:level="3"><nlm:aff id="aff002"><addr-line>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris, France</addr-line>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Carvalho, Celia" sort="Carvalho, Celia" uniqKey="Carvalho C" first="Célia" last="Carvalho">Célia Carvalho</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Carmo Fonseca, Maria" sort="Carmo Fonseca, Maria" uniqKey="Carmo Fonseca M" first="Maria" last="Carmo-Fonseca">Maria Carmo-Fonseca</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
</titleStmt>
<publicationStmt><idno type="wicri:source">PMC</idno>
<idno type="pmid">28742140</idno>
<idno type="pmc">5524367</idno>
<idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5524367</idno>
<idno type="RBID">PMC:5524367</idno>
<idno type="doi">10.1371/journal.pone.0181065</idno>
<date when="2017">2017</date>
<idno type="wicri:Area/Pmc/Corpus">001031</idno>
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<idno type="wicri:Area/Pmc/Curation">001031</idno>
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<idno type="wicri:Area/Pmc/Checkpoint">000722</idno>
<idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Checkpoint">000722</idno>
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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes</title>
<author><name sortKey="Pires, Vanessa Borges" sort="Pires, Vanessa Borges" uniqKey="Pires V" first="Vanessa Borges" last="Pires">Vanessa Borges Pires</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Sim Es, Ricardo" sort="Sim Es, Ricardo" uniqKey="Sim Es R" first="Ricardo" last="Sim Es">Ricardo Sim Es</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Mamchaoui, Kamel" sort="Mamchaoui, Kamel" uniqKey="Mamchaoui K" first="Kamel" last="Mamchaoui">Kamel Mamchaoui</name>
<affiliation wicri:level="3"><nlm:aff id="aff002"><addr-line>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris, France</addr-line>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea>Center for Research in Myology, INSERM UMRS974, CNRS FRE3617, Sorbonne Universités, UPMC Univ Paris 06, Paris</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Carvalho, Celia" sort="Carvalho, Celia" uniqKey="Carvalho C" first="Célia" last="Carvalho">Célia Carvalho</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Carmo Fonseca, Maria" sort="Carmo Fonseca, Maria" uniqKey="Carmo Fonseca M" first="Maria" last="Carmo-Fonseca">Maria Carmo-Fonseca</name>
<affiliation wicri:level="1"><nlm:aff id="aff001"><addr-line>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal</addr-line>
</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa</wicri:regionArea>
<wicri:noRegion>Lisboa</wicri:noRegion>
</affiliation>
</author>
</analytic>
<series><title level="j">PLoS ONE</title>
<idno type="eISSN">1932-6203</idno>
<imprint><date when="2017">2017</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
<profileDesc><textClass></textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en"><p>Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.</p>
</div>
</front>
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<pubmed><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes.</title>
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<author><name sortKey="Carmo Fonseca, Maria" sort="Carmo Fonseca, Maria" uniqKey="Carmo Fonseca M" first="Maria" last="Carmo-Fonseca">Maria Carmo-Fonseca</name>
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<author><name sortKey="Pires, Vanessa Borges" sort="Pires, Vanessa Borges" uniqKey="Pires V" first="Vanessa Borges" last="Pires">Vanessa Borges Pires</name>
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<author><name sortKey="Sim Es, Ricardo" sort="Sim Es, Ricardo" uniqKey="Sim Es R" first="Ricardo" last="Sim Es">Ricardo Sim Es</name>
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<country xml:lang="fr">France</country>
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<region type="old region">Île-de-France</region>
<settlement type="city">Paris</settlement>
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<country xml:lang="fr">Portugal</country>
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<country xml:lang="fr">Portugal</country>
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<series><title level="j">PloS one</title>
<idno type="eISSN">1932-6203</idno>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Cell Line</term>
<term>Dystrophin (biosynthesis)</term>
<term>Dystrophin (genetics)</term>
<term>Exons</term>
<term>Genetic Therapy (methods)</term>
<term>Humans</term>
<term>Muscle Fibers, Skeletal (metabolism)</term>
<term>Muscular Dystrophy, Duchenne (genetics)</term>
<term>Muscular Dystrophy, Duchenne (metabolism)</term>
<term>Muscular Dystrophy, Duchenne (therapy)</term>
<term>Oligonucleotides (genetics)</term>
<term>Oligonucleotides (therapeutic use)</term>
<term>Oligonucleotides, Antisense (genetics)</term>
<term>Oligonucleotides, Antisense (therapeutic use)</term>
<term>Protein Isoforms (biosynthesis)</term>
<term>Protein Isoforms (genetics)</term>
<term>RNA Splicing</term>
</keywords>
<keywords scheme="KwdFr" xml:lang="fr"><term>Dystrophine (biosynthèse)</term>
<term>Dystrophine (génétique)</term>
<term>Exons</term>
<term>Fibres musculaires squelettiques (métabolisme)</term>
<term>Humains</term>
<term>Isoformes de protéines (biosynthèse)</term>
<term>Isoformes de protéines (génétique)</term>
<term>Lignée cellulaire</term>
<term>Myopathie de Duchenne ()</term>
<term>Myopathie de Duchenne (génétique)</term>
<term>Myopathie de Duchenne (métabolisme)</term>
<term>Oligonucléotides (génétique)</term>
<term>Oligonucléotides (usage thérapeutique)</term>
<term>Oligonucléotides antisens (génétique)</term>
<term>Oligonucléotides antisens (usage thérapeutique)</term>
<term>Thérapie génétique ()</term>
<term>Épissage des ARN</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="biosynthesis" xml:lang="en"><term>Dystrophin</term>
<term>Protein Isoforms</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Dystrophin</term>
<term>Oligonucleotides</term>
<term>Oligonucleotides, Antisense</term>
<term>Protein Isoforms</term>
</keywords>
<keywords scheme="MESH" qualifier="biosynthèse" xml:lang="fr"><term>Dystrophine</term>
<term>Isoformes de protéines</term>
</keywords>
<keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Muscular Dystrophy, Duchenne</term>
</keywords>
<keywords scheme="MESH" qualifier="génétique" xml:lang="fr"><term>Dystrophine</term>
<term>Isoformes de protéines</term>
<term>Myopathie de Duchenne</term>
<term>Oligonucléotides</term>
<term>Oligonucléotides antisens</term>
</keywords>
<keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Muscle Fibers, Skeletal</term>
<term>Muscular Dystrophy, Duchenne</term>
</keywords>
<keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Genetic Therapy</term>
</keywords>
<keywords scheme="MESH" qualifier="métabolisme" xml:lang="fr"><term>Fibres musculaires squelettiques</term>
<term>Myopathie de Duchenne</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Oligonucleotides</term>
<term>Oligonucleotides, Antisense</term>
</keywords>
<keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Muscular Dystrophy, Duchenne</term>
</keywords>
<keywords scheme="MESH" qualifier="usage thérapeutique" xml:lang="fr"><term>Oligonucléotides</term>
<term>Oligonucléotides antisens</term>
</keywords>
<keywords scheme="MESH" xml:lang="en"><term>Cell Line</term>
<term>Exons</term>
<term>Humans</term>
<term>RNA Splicing</term>
</keywords>
<keywords scheme="MESH" xml:lang="fr"><term>Exons</term>
<term>Humains</term>
<term>Lignée cellulaire</term>
<term>Myopathie de Duchenne</term>
<term>Thérapie génétique</term>
<term>Épissage des ARN</term>
</keywords>
</textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en">Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to RNA, potent biological activity, high stability and low toxicity in vivo. Here, we designed a series of LNA-based SSOs complementary to two sequences of the human dystrophin exon 51 that are most evolutionary conserved and evaluated their ability to induce exon skipping upon transfection into myoblasts derived from a DMD patient. We show that 16-mers with 60% of LNA modification efficiently induce exon skipping and restore synthesis of a truncated dystrophin isoform that localizes to the plasma membrane of patient-derived myotubes differentiated in culture. In sum, this study underscores the value of short LNA-modified SSOs for therapeutic applications.</div>
</front>
</TEI>
</pubmed>
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